Summer 2020 PMPRB Webinar Series: Webinar 2 (July 16, 2020) Hearing From Those Who Really Matter. This Webinar will take place after the PMPRB’s promised rescheduled “public forums” and “research webinars.” Roundtable Lindy Forte, Principal Consultant, Patient Access Solutions Dr. Shawn Whatley, family physician, Munk Senior Fellow, Macdonald Laurier Institute and past president of the OMA Barbara Jaszewski, Advisor Cloud and past global vice president of pricing and market access Catherine Boivin, SMA Patient Durhane Wong-Rieger, President & CEO, CORD Moderator: Bill Dempster, CEO, 3Sixty Public Affairs

1. Hearing From Those Who Really Matter
Summer Webinar Series on the PMPRB Changes
July 16, 2020
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2. FOR PMPRB
• Lives matter. Explore impact of pricing policies on patients’ lives and build polices to support drugs getting to
Canadian patients.
• PMPRB should act less like a punitive body and more like a “public good” agency. Collaborate with patients,
clinicians, payers, and other stakeholders to arrive at pricing guidelines that work for all.
• If goal is 20% reduction toward OECD median, as announced, implement new 11-country comparison; economic
factors assessed by CADTH/INESSS as starting point for negotiation; do not set PMPRB maximums using $/QALY
FOR POLICY MAKERS
• This next generation of drug are revolutionary. Ensure that Canada’s pharmaceutical policies, including pricing
policies, will allow Canadians to have access as soon as possible.
• STOP the PMPRB … until all of the proper consultations and considerations have been conducted.
• Expand upon COVID-19 Public-Private partnerships between government and pharmaceutical industry. Engage
early in development process; communicate continuously; seek preferred partner pricing.
RECAP ON CORD’S KEY RECOMMENDATIONS

3. The PMPRB HAS updated its draft guidelines – there have been changes, including higher thresholds
and price reduction “floors,” however, the system continues to be formulaic leading to two questions:
1. If these guidelines come into being, will patients benefit or be harmed?
2. Will the guidelines allow for more holistic strategies and alternative approach as we discussed in
the spring series? (amortization, managed access, outcomes-based agreements)
We now have until August 4 to develop written input – and participate in a webinars. CORD wants to
ensure all stakeholders have an opportunity to closely examine how the changes will actually impact
patients, especially regarding timely access to new medicines and clinical trials
The goal of our summer webinar series: understand the PMPRB’s updated draft guidelines, allow an
opportunity for dialogue on the latest changes with officials, and mobilize and coordinate efforts to
communicate our collective input and concerns to all levels of government
CONTEXT FOR PMPRB SUMMER WEBINAR SERIES
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4. • Lindy Forte Patient Access Solutions
• Barbara Jaszewski Advisor Cloud and past global VP of pricing and market access
• Dr. Sandy Sehdev Medical Oncologist at the Ottawa Hospital Cancer Centre
• Catherine Boivin SMA Patient
• Durhane Wong-Rieger Canadian Organization for Rare Disorders
MODERATOR Bill Dempster, 3Sixty Public Affairs
Today’s roundtable
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5. PMPRB Webinars to Date
https://www.canada.ca/en/pat
ented-medicine-prices-
review/services/consultations/
draft-guidelines.html

6. • Held “Research webinars” on the following topics:
• Spending on expensive drugs for rare diseases
• Market size of patented medicines in Canada
• Drug pricing and its impact on R&D investments, clinical trials and
availability of medicines in Canada
• Drug shortages in Canada (at 1:30pm today)
• “Public webinar”
• International examples (use of economic factors)
• Impacts on access to medicines and clinical trials
• Specific call-out on new medicines for cystic fibrosis
• Case studies starting point understates impact of comparator
country changes
What we have heard so far on the webinars
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7. Insight into the Spending on
Drugs for Rare Diseases
in Canada
PMPRB Analysis vs. An Alternate
Point of View
PATIENT ACCESS SOLUTIONS

8. Table of Contents 8
1. Brief overview of the PMPRB report on “Insight into the
spending on expensive drugs for rare diseases”
• What did we hear?
• What did we not hear?
2. Alternate analysis of the projected cost of drugs for rare diseases
(DRDs) in Canada
3. Perspective on the cost of DRDs in Canada relative to other
expenditures
4. Where can we go from here?

9. Overview of the PMPRB Report on EDRD Spending 9
Methods
• Definition of “Expensive” Drugs for Rare Diseases (EDRDs):
• at least one orphan designation through either FDA or EMA, and
• costs > $100,000 per patient per year for non-oncology drugs, or
• > $7,500 per 28 days for oncology drugs.
• Annual per patient costs obtained from the Canadian Agency for Drugs and Technologies in
Health (CADTH) (assumed)
• Total annual cost of claims for these drugs were obtained from the IQVIA database (2009-2019)

10. What does the report say?
• “Orphan medicines are increasingly dominating the new drug
landscape…”
• “EDRDs [are] the fastest growing market segment, pushing the
limits of affordability”
• “…most offer limited or unclear therapeutic benefit
• “…not cost-effective at their list price”
• “EDRD spending in Canada is above the OECD norm”
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11. “EDRDs are the fastest growing market segment” 11
From: PMPRB Research Webinar. Insight into the spending on expensive drugs for rare diseases. June 23, 2020. Page 10.

12. What does the report not say? 12
PMPRB Report Missing Perspective
“Orphan medicines are
dominating the
landscape”
• Comments on innovation; cause of the diseases were previously untreatable
• Patient/caregiver perception of value as life changing or life extending
• DRDs (42) and targeted cancer drugs (51) are bringing the greatest incremental
health gains to Canadian patients
“…pushing the limits of
affordability”
• Costs of the 42 DRDs account for only 2.5% of the total pharmaceutical sales
• Private insurance covers 40% or more of the costs even when publicly reimbursed
• Actual prices paid are much lower than the list prices due to confidential
agreements
• Payers are involved in the collaborative decision regarding the net price
• Mechanisms are in place to ensure value is achieved and the risks of exceeding
cost projections are minimized
• Annual cost of DRDs (~$280M) pales in comparison with costs of smoking-related
illness and obesity - $6.5B and $8.8B (for 2021), respectively

13. What does the report not say? (con’t) 13
PMPRB Report Missing Perspective
“…limited or unclear
therapeutic benefit”
• The drugs are approved by Health Canada through Priority Review mechanisms
due to lack of available treatment alternatives
• Low patient numbers are due to rarity of the diseases
• Lack of transparency in the PMPRB therapeutic designation
• Lack of consistency between research reports (Board Staff/Drug Information
Centre) designating drugs as “breakthrough” vs recommendations that are
downgraded by the expert committee (HDAP)
“…spending…is above
the OECD norm”
• Canada’s expenditure appears to be at or below the norm when looking at
orphan drugs only
• Context of heredity of rare disease and our more heterogeneous population; is
expenditure in line with proportion of population afflicted?

14. Alternate analysis of DRD costs in Canada 14
• Patient Access Solutions undertook an analysis of the current and future budget impact of
DRDs in Canada; presented at the 2019 International Society for Pharmacoeconomics and
Outcomes Research (ISPOR) Meeting in Copenhagen
• Methods:
• The cost of DRDs was included while cancer drugs were excluded
• DRDs are used lifelong; oncology drugs are used for a more limited duration of treatment
• CADTH has recognized the differences between oncology and non-oncology drugs and
reviews the drugs through different pathways
• Cancer drugs usually are approved for more than one indication and/or more than one
line of therapy superseding the initial “orphan” designation
• The prevalence of cancers typically increases over time while rare diseases are genetic
aberrations and rates of inheritance are more stable

15. Alternate analysis – methods (con’t)
• Our analysis included:
• Only the expected public drug program expenditures
• Private insurers in Canada are providing DRD benefits to employees
• Health benefits are seen as a means to ensure employees and caregivers are maximally
productive at work and employees are valued
• Private insurers are mobilizing to pool risks to ensure their ability to continue to provide DRDs
• The cost of drugs not available in the claims dabatase - obtained through FOI requests
• The cost of non-”expensive” drugs (i.e., drugs with annual costs <$100k)
15

16. The total public expenditure on DRDs reached
$280 million in 2019
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17. The total expenditure on DRDs was ~2%
of the total drug budget in 2019
17
2%
2020
4%
2022
5%
2023
6%
2024
6%
2025

18. The expenditure on DRDs is orders of magnitude
less than other expenditures valued by Canadians
18

19. 19

20. Where can we go from here?
• Acknowledge that we need to collaborate to find a way to ensure that our fellow Canadians
can continue to have access to the drugs that provide the best possible health, physical
function, and lifespan possible despite being afflicted with a rare disease
• Develop a multi-stakeholder consensus on the methods that should be employed in the
realistic projection of future DRD costs to ensure fiscal predictability and continued funding
of DRDs where clinical value is offered
• Find creative ways to ensure Canadians’ long-term ability to fund life changing treatments
for patients, e.g.,
• Disinvest: avoid waste, duplication of effort, funding of ineffective technologies, etc
• Pool risk across jurisdictions
• Ensure that Canada’s ideals of universal healthcare and caring for all are upheld in
regulations/practices ensuring that Canadians with rare diseases are not further
disadvantaged compared to the rest of our society and compared with citizens of other
(OECD) countries
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21. JULY
16
Federal Price Controls and Access
to Medicines Webinar Series
JUNE
25
JULY
30
Has the PMPRB removed
the obstacles to access
in its Guidelines?
at noon at noon at noon
Hearing From
Those Who Matter
Collaborating on Drug
Pricing Guidelines for Cost-
Effectiveness and Access